Pharmaceutical Technology Editors

The European Commission (EC) has granted Vico Therapeutics with orphan drug designation for its investigational antisense oligonucleotide therapy (VO659) for the treatment of Spinocerebellar Ataxia (SCA).

According to a Feb 10, 2021 press release, the orphan designation was granted based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. A previous orphan designation has also been granted for VO659 for the treatment of Huntington Disease.

“[SCA] make up a great part of the so called polyglutamine disorders, debilitating and progressive diseases, leading to significant impairment of mobility, speech, and multiple other daily activities of patients suffering from this condition. So far, there is no treatment which can halt the progression of these diseases,” said Rupert Sandbrink, chief medical officer at Vico, in the press release.“Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases. We’re pleased to receive orphan drug designation, which recognizes both the unmet medical need for patients living with spinocerebellar ataxias, and the potential of our approach.”

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The EC has granted Vico Therapeutics with orphan drug designation for its investigational antisense oligonucleotide therapy for the treatment of SCA.



EC Grants Orphan Drug Designation to Vico Therapeutics’ Oligonucleotide SCA Therapy

Artificial Intelligence (AI) drug discovery company, Exscientia, has formed a partnership with the Alzheimer’s Research United Kingdom University of Oxford Drug Discovery Institute (ARUK-ODDI) for the development of medicines targeting neuroinflammation to treat Alzheimer’s disease (AD).

The partnership, which was announced in a Feb. 11, 2021 press release, will combine Exscientia’s AI-driven molecular design capabilities with the deep therapy area knowledge and technical expertise of the ARUK-ODDI. The collaborative efforts will focus on a specific neuroinflammatory pathway implicated in the development of AD—activation of the NLRP3 inflammasome.

“Alzheimer’s is a dreadful disease that affects tens of millions worldwide. Despite clinical trials of numerous agents over a wide range of mechanisms, the last new Alzheimer’s medication, was approved nearly two decades ago,” said Exscientia’s Chief Operating Officer David Hallett, in the press release. “Alzheimer’s drug development is costly, complex, and extremely challenging with clinical trial failure rate being the highest of any therapeutic area. Our mission is to make novel drugs available to all and we are excited to utilize our AI drug discovery platform and work alongside the expertise of the [ARUK-ODDI] team to accelerate innovation and develop potential medicines to solve this global epidemic.”

“We are delighted to be partnering with Exscientia. Their state-of-the-art AI capabilities will enable us to investigate multiple molecules in parallel and accelerate the project towards candidate declaration,” added Dr. John Davis, chief scientific officer of the ARUK-ODDI, in the press release. “Human genetic variation points towards a critical role for the body’s immune system in an individual’s risk of developing Alzheimer’s disease. It is vital that we develop treatments that target neuroinflammatory mechanisms underlying dementia.”

Exscientia has formed a partnership with the ARUK-ODDI for the development of medicines targeting neuroinflammation to treat Alzheimer’s disease.

Exscientia Partners with University of Oxford on Alzheimer’s Treatments

Gilead announced on Feb. 9, 2021 that it is opening a business services center in the Research Triangle region of North Carolina.

The new site is set to be fully operational by the third quarter of 2021 and will house the company’s North American human resource operations, along with certain finance and information technology teams, Gilead said in a company press release. In addition, the site will be staffed by 275 employees.

“The creation of the Gilead Business Services center is an important step forward for the company, as it creates a dedicated hub for critical support services in a location that will allow us to realize certain cost and time zone advantages,” the company added in the press release.

The new business services site will house the company’s North American human resource operations, along with certain finance and information technology teams.

Gilead to Open New Location in North Carolina

Eli Lilly announced on Feb. 9, 2021 that FDA has granted emergency use authorization (EUA) to bamlanivimab (LY-CoV555) and etesevimab (LY-CoV016), Eli Lilly’s antibody cocktail for the treatment of mild to moderate COVID-19 in patients ages 12 and older who are at high risk for progressing to severe COVID-19 or hospitalization.

The EUA is based on positive results from a Phase III trial that showed bamlanivimab and etesevimab together reduced the risk of COVID-19 hospitalizations and death by 70%, Eli Lilly said in a company press release. The antibodies are administered via a single intravenous infusion after a positive COVID-19 test and within 10 days of symptom onset.

"Lilly has dedicated our time, resources, and expertise to discover and develop therapies to treat COVID-19," said Daniel Skovronsky, MD, PhD, Eli Lilly's chief scientific officer and president of Lilly Research Laboratories, in the press release. "Bamlanivimab alone under emergency use authorization has already provided many people with an early treatment option that could prevent hospitalizations and we are excited to now add an additional therapeutic option with a similar demonstrated clinical benefit. Additionally, with the risk of resistance emerging as various strains of the virus arise, bamlanivimab and etesevimab together could potentially allow efficacy against a broader range of naturally occurring SARS-CoV-2 variants as these new strains spread around the world."

The EUA is based on positive results from a Phase III trial that showed bamlanivimab and etesevimab together reduced the risk of COVID-19 hospitalizations and death by 70%.

 FDA Grants Emergency Use Authorization to Eli Lilly COVID-19 Antibody Cocktail

The European Medicines Agency (EMA) has issued a statement, clarifying its position on the European approval process of the Sputnik V vaccine, which is being developed by the Gamaleya National Centre of Epidemiology and Microbiology in Russia.

According to a Feb. 10, 2021 press release, the regulatory body has asserted that, despite reports stating the contrary, it has not received an application for the rolling review or a marketing authorization of Sputnik V. EMA has provided the vaccine’s developers with scientific advice and is in dialogue with the developers regarding next steps.

Sputnik V developers have expressed an interest in submitting the vaccine for rolling review consideration—a process that can only be employed during emergency situations. However, EMA’s Committee for Medicinal Products for Human Use needs to agree that the vaccine is suitable to progress via the accelerated review procedure prior to submission of the application.

Furthermore, EMA emphasized in the statement that any new assessments of COVID-19 vaccines or medicines being undertaken by the agency would be promptly announced so that the public are informed. Therefore, if there has been no communication from EMA regarding a vaccine or medicine, then the status of that specific therapy remains unchanged.

EMA has clarified its position on the European approval process of the Sputnik V vaccine. 

EMA Clarifies Position on Sputnik V European Approval Process

Evonik has revealed plans for investment in the short-term expansion of its specialty lipids production, which are used with mRNA-based COVID-19 vaccines. The announcement was made in a Feb. 11, 2021 press release.

The company’s sites in Hanau and Dossenheim (Germany) will start producing commercial quantities of lipids from the second half of 2021 as a part of a strategic partnership with vaccine manufacturer BioNTech. Through this latest investment, the strategic partnership between Evonik and BioNTech will be strengthened and the supply security of the Pfizer-BioNTech vaccine will be increased.

“The pandemic requires decisive action,” said Christian Kullmann, chairman of Evonik’s executive board, in the press release. “We are therefore doing everything possible to supply our partners with the critical lipids they need. At the same time, we are expanding our production capacity and competencies along the entire value chain.”

“With our partnership with BioNTech, we are systematically expanding our leading position as an integrated development partner in cell and gene therapies,” added Thomas Riermeier, head of Evonik's Health Care business line, in the press release. “Evonik’s portfolio includes pharmaceutical excipients such as lipids, as well as CDMO services for the formulation development, GMP manufacturing, and aseptic filling of complex parenteral drug products.”

Evonik has revealed plans for investment in the short-term expansion of its specialty lipids production, which are used with mRNA-based COVID-19 vaccines. 